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2. Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1. Chandler RJ; Williams IM; Gibson AL; Davidson CD; Incao AA; Hubbard BT; Porter FD; Pavan WJ; Venditti CP Hum Mol Genet; 2017 Jan; 26(1):52-64. PubMed ID: 27798114 [TBL] [Abstract][Full Text] [Related]
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